Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
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A new drug intended to make it easier to clear mucus from the lungs of people with cystic fibrosis showed promising results in a Phase 2 clinical trial. The drug, called SPX-101, blocks the overactive sodium channels that line CF lung cells.
The top priority in clinical research is protecting the safety of people who participate. Learn more about the numerous layers of review that help keep participants safe.
Cystic Fibrosis Foundation Therapeutics Inc. made its first major award last month to advance gene editing research in cystic fibrosis. Editas Medicine Inc. will explore the potential of using what some scientists refer to as “a tiny pair of scissors” to snip out the genetic defect that causes CF.
Dr. John P. Clancy shares new developments in drugs that restore airway surface liquid in the lungs of people with cystic fibrosis, making it easier to clear mucus.
The Cystic Fibrosis Foundation is funding research into gene editing techniques to see if they can be used to fix the mutations that cause cystic fibrosis. One of the most popular techniques is CRISPR-Cas9. To see how this might work for CF, watch this video.
Many people might be put off by the thought of participating in a clinical trial. The increasing life expectancy for CFers like me, however, is a testament to the impact medical research can have on the development of therapies for cystic fibrosis.
Drs. Drucy Borowitz and Manu Jain share new developments in drugs that will treat the underlying cause of cystic fibrosis.
Historic Time in Treatment of Rare Disease as Promising Drugs Reach Phase 3 Trials